HOPE Clinical Trial Provides Hope for Duchenne Families

CureDuchenne is pleased to see the positive preliminary data from Capricor Therapeutics on their HOPE clinical trial in Duchenne muscular dystrophy. CureDuchenne Ventures helped fund this clinical trial to address cardiac failure, the primary cause of death for Duchenne patients.  This data provides hope to  families with Duchenne.

Capricor Therapeutics announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne muscular dystrophy. Patients treated with CAP-1002 demonstrated statistically-significant improvements in measure of cardiac and upper limb function.

CureDuchenne continues to play an instrumental role in finding, funding and fostering a pipeline of Duchenne research that advance to clinical trials and leads to treatments such as Exondys51.  CureDuchenne identifies good science and funds research early on to help advance drug development to help treat the whole disease.  CureDuchenne has funded exon skipping, gene editing, therapy, anti-inflammatory, anti-fibrotic as well cardiac treatments.

Thank you to donors who contributed to the Capricor research.

Our sincere gratitude to the Duchenne patients who participated in the HOPE clinical trial.  We appreciate your selfless sacrifice to help move the science forward so others can benefit.

We will continue to accelerate drug development and fund promising treatments until there is a cure for all those with Duchenne.